Article ID Journal Published Year Pages File Type
8644393 Gene 2018 27 Pages PDF
Abstract
CRISPRClustered Regularly Interspaced Short Palindromic RepeatsCasCRISPR-associatedERTenzyme replacement therapyHDRhomology-directed repairHSCThematopoietic stem cell transplantationIDUAalpha-L-iduronidaseLSDlysosomal storage disorderMPS IMucopolysaccharidosis type I
Keywords
Related Topics
Life Sciences Biochemistry, Genetics and Molecular Biology Genetics
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CRISPR-Cas9-mediated gene editing in human MPS I fibroblasts
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